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uniQure Announces Enrollment of First Two Patients in Second Cohort of Phase I/II Clinical Trial of AMT-130 for the Treatment of Huntington’s Disease
LEXINGTON, Mass. and AMSTERDAM, The Netherlands, June 16, 2021 (GLOBE NEWSWIRE) -- uniQure N.V. (NASDAQ: QURE), a leading gene therapy company advancing
About this update from Uniqure N.v.
[{"type":"text","content":"LEXINGTON, Mass. and AMSTERDAM, The Netherlands, June 16, 2021 (GLOBE NEWSWIRE) -- uniQure N.V. (NASDAQ: QURE), a leading gene therapy company advancing transformative therapies for patients with severe medical needs, today announced that the first two patient procedures have been completed in the higher dose, second cohort of the Phase I/II clinical trial of AMT-130 for the treatment of Huntington’s disease. The initiation of patient enrollment in this 16-patient cohort follows a meeting last month of the trial’s independent Data Safety Monitoring Board (DSMB) that reviewed safety data for the fully enrolled first cohort of ten patients. The Phase I/II study is a double-blind, randomized clinical trial being conducted in the United States. To date, seven patients now have been treated with AMT-130 across both trial cohorts, and five patients have received the imitation surgery. “We are very pleased with the progress that we continue to make in this Phase 1-2 clinical trial and that we are now administering AMT-130 at the higher dose,” said Ricardo Dolmetsch, Ph.D., president of research and development at uniQure. “We look forward to ongoing patient enrollment in the second cohort and to initiating clinical development of AMT-130 in Europe in the second half of this year. We also remain on track to share initial imaging and biomarker data from the U.S. clinical trial before the end of the year.” About the Phase I/II Clinical Program of AMT-130 The U.S. Phase I/II clinical trial of AMT-130 for the treatment of Huntington’s disease will explore the safety, tolerability, and efficacy signals in 26 total patients with early manifest Huntington’s disease split into a 10 patient, low-dose cohort followed by a 16 patient, higher-dose cohort randomized to treatment with AMT-130 or an imitation (sham) surgery. The multi-center trial consists of a blinded 12-month core study period followed by unblinded long-term follow-up for five years. Patients will receive a single administration of AMT-130 through MRI-guided, convection-enhanced stereotactic neurosurgical delivery directly into the striatum (caudate and putamen). Additional details are available on www.clinicaltrials.gov (NCT04120493). The European, open-label Phase Ib/II study of AMT-130 will enroll 15 patients with early manifest Huntington’s disease across two dose cohorts. Toget...